Department of Pediatric Oncology/Hematology of the VU University Medical Center, De Boelelaan 1117, NL-1081, HV Amsterdam, The Netherlands.
Expert Rev Anticancer Ther. 2012 Mar;12(3):405-13. doi: 10.1586/era.12.1.
Pediatric acute myeloid leukemia (AML) is currently associated with survival rates as high as 70%. However, many events still occur, side effects are significant, and late effects occur and can even be life-threatening. Thus, the treatment of pediatric AML still needs further improvement. While most study groups agree on several principles of AML treatment, many unanswered questions and even controversies remain, which will be the topic of this review. Relapsed AML, the most frequent event in children, will also be discussed. The controversies justify future clinical studies. Fortunately, biotechnical developments provide novel treatment targets and targeted drugs, and will enable minimal residual disease-driven tailored therapy. Moreover, a wide range of new drugs is being developed. International collaboration is required to perform randomized, or even single-arm clinical studies, in this setting of subgroup-directed therapy, and fortunately is being accomplished. Therefore, optimism is justified and the treatment of pediatric AML will continue to improve.
儿科急性髓细胞白血病 (AML) 目前的生存率高达 70%。然而，仍有许多事件发生，副作用显著，且迟发性副作用会出现，甚至危及生命。因此，儿科 AML 的治疗仍需进一步改善。虽然大多数研究组对 AML 治疗的几个原则达成了一致，但仍有许多未解决的问题，甚至存在争议，这将是本次综述的主题。复发的 AML 是儿童中最常见的事件，也将进行讨论。这些争议为未来的临床研究提供了依据。幸运的是，生物技术的发展为新的治疗靶点和靶向药物提供了可能，并将使基于微小残留病灶的个体化治疗成为可能。此外，正在开发多种新药。在这种亚组定向治疗的情况下，需要进行国际合作来开展随机或甚至单臂的临床研究，而这也正在实现。因此，有理由保持乐观，儿科 AML 的治疗将继续改善。
